News

The FDA has granted Fast Track Designation for Cyprium Therapeutics’ CUTX-101, developed for the treatment of certain patients with classic Menkes disease.

In these new study results, patients administered 300 mg eptinezumab reported 8.2 fewer MMDs from baseline—when mean patient MMDs were 16.1 per month. The rate was significantly improved from that of patients administered placebo.

New research finds patients with HIV and chronic pain are aware that opioids are addictive, yet most don’t receive common addiction-prevention monitoring.

One year after the third and fourth quarterly infusions, those administered the 300-mg dose of eptinezumab reported reductions of 5.2 monthly migraine days, compared to 4.0 days for patients in the placebo group.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

These regimens can also ensure the maintenance of visual acuity levels that can keep patients self-sufficient in real-world scenarios, such as driving, reading, and recognizing faces.

The FDA has cleared commencement for Retrotope’s open-label phase 2/3 clinical trial of its investigational drug RT001 to evaluate its efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD).

The LIBERATE study revealed 47.7% of those treated with the Zephyr Endobronchial Valve experienced at least a ≥15% improvement on pulmonary function scores, compared with 16.8% of the control group.

Positive developmental updates on the investigational BTK inhibitor, zanubrutinib, as a treatment for Waldenström macroglobulinemia were recently released.

A study of low-income mothers found that participants who experienced problem-solving sessions reported 11% lower stress compared to controls.

Researchers from McGill University say they’ve found genetic variants in patients with childhood-onset schizophrenia, offering a new direction for the development of treatment drugs.

Sterile syringe access as part of comprehensive HIV prevention is important to control and prevent outbreaks.

The FDA has granted orphan drug designation to Oncology Venture AB for their oral phase 3 multi tyrosine kinase inhibitor (previously TKI258), dovitinib DRP, for the treatment of adenoid cystic carcinoma (AdCC).

Multiple hematology organizations have come together to develop state-of-the-art guidelines for the diagnosis and management of von Willebrand Disease.

The supplement included data from decade-long noninferiority PRECISION trial, which revealed safety data for using celecoxib in conjunction with aspirin.

Orphan drug designation has been granted to CPI-613 for the treatment of Burkitt lymphoma as a phase 2 trial commences for the drug.

The new treatment against multidrug-resistant Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae (CRE), just received US Food and Drug Administration (FDA) approval.

In an invited commentary, a trio of authors called the concept of improving or protecting visual function as a viable intervention to prevent or control cognitive decline overall both “plausible and attractive.”

Individuals with vitamin D deficiency may be at greater risk of interstitial lung disease, a rare disease that causes scarring of the lungs.

People living with mental illness face great adversity. One of their greatest challenges is the seemingly intractable problem of stigma.

The US Food and Drug Administration (FDA) has approved encorafenib and binimetinib (BRAFTOVI and MEKTOVI) in combination for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation as detected by an FDA-approved test.

Positive top-line data from Part A of a recent phase 3 trial evaluating voxelotor for the treatment of sickle cell disease have been released.

Updated data on MoveDMD, a phase 2, open-label extension trial of edasalonexent in boys diagnosed with Duchenne muscular dystrophy (DMD), reports positive results.

If approved, REMOXY ER will be classified as a schedule II drug and would be subject to strict prescribing and dispensing rules.

New research from the University of Virginia shows that a smartphone app can make a major impact on the health of people living with HIV.

Just 12% of the US population, African-Americans account for nearly 50% of the HIV/AIDS cases in the country.

Takami Sato, MD, PhD, Department of Medical Oncology, Kimmel Cancer Center, Thomas Jefferson University, discusses new treatment for metastatic uveal melanoma: IMCgp100.

Rigel Pharmaceuticals has recently selected US Bioservices to dispense fostamatinib disodium hexahydrate tablets, for the treatment of adult patients with chronic immune thrombocytopenia (ITP).

New findings suggest that riluzole prolongs survival in the final clinical stage of amyotrophic lateral sclerosis (ALS).

If approved, this would be the first oral, 1-dose antiviral and the first medicine with a novel proposed mechanism of action to treat the flu in about 20 years.